DISGENET is provided by Medbioinformatics Solutions S.L.
Rambla de Cataluña 14, 7º 1ª 08007 Barcelona (Spain)
Contact us at

logo

Introduction

Target selection remains a key determinant of success in pharmaceutical R&D, as efficacy and safety issues continue to drive late-stage failures. In 2025, the FDA approved 46 drugs (Mullard 2026) (Table 1), including 38 protein-targeted therapies and three mRNA-based treatments, offering a real-world snapshot of which targets actually make it to the finish line. By integrating data on disease associations from DISGENET (Piñero et al. 2026), gene expression, and historical target status, we explored what these approvals can teach us about choosing the right targets.

From Legacy Targets to Emerging Opportunities

All drug targets are represented in DISGENET, underscoring their established relevance to human disease. Most are associated with hundreds of disease phenotypes. All are referenced in hundreds, often thousands, of publications in the disease genomics literature, reflecting decades of accumulated biological and clinical knowledge. Consistent with this depth of evidence, many 2025 FDA approvals focus on long-studied targets such as EGFR, ERBB2, and ESR1, which exhibit publication histories dating back to the 1970s and broad links to diverse disease areas.

Translational and Disease Insights

DISGENET also shows that most of the approved targets appear in clinical trial contexts. Examples include patient stratification (e.g., JAK2, MET) and monitoring pharmacodynamic response (e.g., APOC3, PCSK9, BTK), reflecting their translational relevance throughout drug development.

References

Mullard, Asher. 2026. “2025 FDA Approvals.” Nature Reviews Drug Discovery 25 (2): 81–87. https://doi.org/10.1038/d41573-026-00001-z.
Piñero, Janet, Javier Corvi, Natalia Rykova, Anna Guillem, Amelia Martı́nez, Jaione Telleria Zufiaur, Ivo Rivetta, et al. 2026. “DISGENET: Accelerating Data-Driven Discovery in Disease Genomics and Therapeutic Development.” bioRxiv. https://doi.org/10.64898/2026.01.05.697749.